Safe and potent isn’t enough

05.11.2012 - The European Commission declined a market authorisation for the orphan drug Elelyso in spite of a positive risk-benefit assessment.

The drug couldn’t be approved due to specific requirements of the European Union (EU) Orphan Drug Regulation. Pfizer Inc. and Israel’s Protalix BioTherapeutics, Inc. developed taliglucerase alfa, as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease. They filed in November 2010 for marketing approval in the EU. In June 2012 the CHMP issued its opinion on the plant cell-expressed recombinant form of human glucocerebrosidase, stating that it could not recommend the drug for approval, because Irish drugmaker Shire’s velaglucerase alfa had received prior Marketing Authorisation with orphan drug designation for the same condition. Therefore, Shire's treatment has orphan market exclusivity in the EU for a ten-year period commencing on its authorisation in August 2010. Pfizer pursued a request for derogation from Shire's orphan market exclusivity based on a number of factors. In detail, Pfizer had been seeking an exemption to orphan exclusivity on the basis of an "insufficient supply" of enzyme replacement therapy for Gaucher's disease and the drug’s expected "major contribution to patient care." But the reasoning of the pharma giant was not enough to convince the CHMP. In USA, Australia, and Japan, Genzyme's orphan drug Cerezyme dominates the Morbus Gaucher market.

"We are disappointed by the EC's decision on taliglucerase alfa and believe it is important, given the history of past shortages, for the Gaucher disease community in the EU to have a third treatment option available," said Diem Nguyen, General Manager, Pfizer Biosimilars. "We will continue to work closely with our partner, Protalix, to make taliglucerase alfa available to the Gaucher disease community in other countries." Taliglucerase alfa was approved by the FDA in May 2012 for the long-term ERT of adults with a confirmed diagnosis of Type 1 Gaucher disease and was approved by Israel's Ministry of Health in September 2012.

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